FIELD: biotechnology.
SUBSTANCE: invention relates to biotechnology, specifically designed synthetic micro-RNAs directed to BCL11A for RNA polymerase II expression, and methods of using them for treating haemoglobinopathies, such as sickle cell anaemia or thalassemia by increasing levels of foetal haemoglobin expression.
EFFECT: invention discloses compositions and methods for treating haemoglobinopathies.
13 cl, 57 dwg, 14 ex
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